Patent SG-11201808912Q-A (SG)
Title: Targeted nucleic acid conjugate compositions
Inventor: HEYES JAMES
IPC: C12N15/87
Filed: 2017-04-11
Abstract: WO 17 / 177 3 26 Al (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/177326 Al 19 October 2017 (19.10.2017) WIPO I PCT (51) International Patent Classification: C12N 15/113 (2010.01) CO7D 209/52 (2006.01) A61K 47/54 (2017.01) C07F 9/40 (2006.01) CO7C 217/52 (2006.01) CO7H 15/26 (2006.01) CO7C 235/06 (2006.01) CO7H 21/00 (2006.01) CO7C 237/08 (2006.01) CO7H 21/02 (2006.01) CO7C 237/20 (2006.01) C07J 63/00 (2006.01) CO7D 207/08 (2006.01) C07J 9/00 (2006.01) CO7D 207/12 (2006.01) Cl 2N 15/87 (2006.01) CO7D 209/12 (2006.01) (21) International Application Number: PCT/CA2017/050447 (22) International Filing Date: 11 April 2017 (11.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/321,034 11 April 2016 (11.04.2016) US 62/417,156 3 November 2016 (03.11.2016) US 62/438,310 22 December 2016 (22.12.2016) US (71) Applicant: PROTIVA BIOTHERAPEUTICS, INC. [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). (72) Inventors; and (71) Applicants : HEYES, James [CA/CA]; 100-8900 Glenly- on Parkway, Burnaby, British Columbia V5J 5J8 (CA). HOLLAND, Richard J. [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). MARTIN, Alan D. [CA/CA]; 100-8900 Glenlyon Park- way, Burnaby, British Columbia V5J 5J8 (CA). WOOD, Mark [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). (74) Agent: SMART & BIGGAR; PO Box 11115, #2300- 1055 West Georgia Street, Vancouver, British Columbia V6E 3P3 (CA). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: — with international search report (Art. 21(3)) [Continued on next page] (54) Title: TARGETED NUCLEIC ACID CONJUGATE COMPOSITIONS 1 11111 11111111 0 111 0 1 11111 11111 0 10 1111 I 1 1 1 0 10 1110 11111 0 111 0 111 1111 1111111 1111 0 11 1111 HN '10° 0 O H OAc NH AGO O NH X d Figure 1 (57) : The invention provides conjugates that comprise a targeting moiety, a nucleic acid, and optional linking groups as well as synthetic intermediates and synthetic methods useful for preparing the conjugates. The conjugates are useful to target thera- peutic nucleic acids to the liver and to treat liver diseases including hepatitis (e.g. hepatitis B and hepatitis D). WO 2017/177326 Al MIDEDIMOM0101011101010MOIDEHIIIMMEMOVOIMIE before the expiration of the time limit for amending the — with sequence listing part of description (Rule 5.2(a)) claims and to be republished in the event of receipt of amendments (Rule 48.2(h))